Non-randomised trial of a lipid lowering drug and a steroid for the treatment of acute myeloblastic leukaemia
| ISRCTN | ISRCTN50635541 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN50635541 |
| Secondary identifying numbers | N/A |
- Submission date
- 03/07/2008
- Registration date
- 25/07/2008
- Last edited
- 28/09/2011
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Cancer
Prospectively registered
Protocol
Statistical analysis plan
Results
Individual participant data
Plain English summary of protocol
Not provided at time of registration
Contact information
Dr Mark Drayson
Scientific
Scientific
Clinical Immunology and Division of Immunity and Infection
The Medical School
University of Birmingham
Edgbaston
Birmingham
B15 2TT
United Kingdom
Study information
| Study design | Interventional multicentre non-randomised phase II study |
|---|---|
| Primary study design | Interventional |
| Secondary study design | Non randomised controlled trial |
| Study setting(s) | Hospital |
| Study type | Treatment |
| Scientific title | The use of Bezfibrate and medroxyProgesterone acetate in Acute Myeloid Leukaemia and refractory anaemia with excess of blasts (RAEB) type 2: a phase II non-randomised trial |
| Study acronym | BaP in AML |
| Study objectives | That patients with acute myeloblastic leukaemia (AML) who would not otherwise receive anti-leukaemia therapy will respond to therapy with bezafibrate and medroxyprogesterone acetate. |
| Ethics approval(s) | Ethics approval received from: 1. The South Birmingham Research Ethics Committee on the 9th April 2003 (ref: 5355) 2. University Hospitals Coventry and Warwickshire Research and Development Department on the 7th July 2004 (ref: NJ02/0304/EU) 3. The Research Ethics Committee of Glasgow Royal Infirmary on the 24th July 2003 (ref: 03HA010) |
| Health condition(s) or problem(s) studied | Elderly and relapsed high risk acute myeloid leukaemia |
| Intervention | 1. Bezafibrate (Bezalip-Mono) 400 mg daily 2. Medroxyprogesterone acetate (Provera) 200 mg twice daily Patients will also be given a prophylactic vitamin supplement so that they are not deficient in vitamins A and D, multivitamin tablet containing minimum vitamin A 4000 units and vitamin D 400 units. Treatment was for 18 weeks. All patients were followed up to death (range of follow up was 8 days to 102 weeks from trial entry); one patient is still alive. |
| Intervention type | Drug |
| Pharmaceutical study type(s) | |
| Phase | Phase II |
| Drug / device / biological / vaccine name(s) | Bezafibrate, medroxyprogesterone acetate |
| Primary outcome measure | Tumour response as measured by: 1. Full blood count 2. Transfusion dependency (frequency of red blood cells/platelet transfusions) 3. Percentage blasts in bone marrow and peripheral blood pre and post BaP therapy 4. Bone marrow morphology as determined by blood smears Response will be assessed using Southwest Oncology Group (SWOG) criteria as modified from National Cancer Institute (NCI-) sponsored workshop guidelines. 1. Complete response (CR): less than 5% blasts in a marrow of sufficient cellularity with a peripheral neutrophil count greater than 1 x 10^9/l and platelet count of greater than 100 x 10^9/l determined by two evaluations not less than 4 weeks apart 2. Partial response (PR): as determined by two evaluations not less than 4 weeks apart: 2.1. In RAEB type 2 bone marrow should show greater than 50% decrease in myeloblasts, but not necessarily disappearance of marrow dyspoiesis. In peripheral blood, greater than 50% reduction in deficit from minimum normal levels (UHB haematology reference range) of the haemoglobin, neutrophil and platelet counts (if abnormal at baseline) with an absence of myeloblasts in the peripheral blood. 2.2. In AML bone marrow should show less than 15% myeloblasts with a decrease but not necessarily a disappearance of marrow dyspoiesis with an absence of Auer rods. Plus in peripheral blood there should be a greater than 50% reduction in deficit from minimum normal levels (UHB haematology reference range) of haemoglobin, neutrophil and platelet counts (if abnormal at baseline) with absence of myeloblasts in the peripheral blood. 3. Minor response (MR): decrease in frequency of infections or bleeding episodes and a 50% decrease in transfusion requirements, decrease of marrow dyspoiesis and improvement in peripheral counts but not enough to qualify for PR or CR nor progressive disease can be established 4. No change: neither the criteria for CR, PR, MR nor progressive disease can be established 5. Progressive disease: evidence of increased blasts in bone marrow or peripheral blood |
| Secondary outcome measures | No secondary outcome measures |
| Overall study start date | 01/06/2003 |
| Completion date | 01/04/2006 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Adult |
| Sex | Both |
| Target number of participants | 20 |
| Key inclusion criteria | 1. Patient has acute myeloid leukaemia (this can be any type of de novo or secondary AML, except acute promyelocytic leukaemia), or 2. Patient has refractory anaemia with an excess of blasts (greater than 10%) RAEB type 2 World Health Organization (WHO) criteria 3. Adult patients, either sex |
| Key exclusion criteria | 1. Patient has acute promyelocytic leukaemia 2. Intensive chemotherapy is considered a suitable option 3. Low dose cytotoxic chemotherapy is likely to be required to control a rising blast cell count in the next month 4. Patient has a concurrent active malignancy 5. Patient has uncontrolled systemic disease (e.g. hypertension, diabetes) or severe cardiovascular disease 6. Patient is pregnant or lactating, or are potentially fertile (both males and females) and have not agreed to take adequate contraceptive precautions during the trial 7. Patient aged under 18 years |
| Date of first enrolment | 01/06/2003 |
| Date of final enrolment | 01/04/2006 |
Locations
Countries of recruitment
- England
- United Kingdom
Study participating centre
Clinical Immunology and Division of Immunity and Infection,
Birmingham
B15 2TT
United Kingdom
B15 2TT
United Kingdom
Sponsor information
University Hospital Birmingham NHS Foundation Trust (UK)
Hospital/treatment centre
Hospital/treatment centre
Trust Headquarters
PO Box 9551
Main Drive
Queen Elizabeth Medical Centre
Edgbaston
Birmingham
B15 2PR
England
United Kingdom
| Website | http://www.uhb.nhs.uk/ |
|---|---|
"ROR" |
https://ror.org/014ja3n03 |
Funders
Funder type
Government
The University Hospital Birmingham NHS Foundation Trust (UK) - paying incidental costs
No information available
Results and Publications
| Intention to publish date | |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Not provided at time of registration |
| Publication and dissemination plan | Not provided at time of registration |
| IPD sharing plan |
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| Results article | results | 01/04/2010 | Yes | No |
